Rising to the Challenge: Meeting a Significant Therapeutic Need

Approximately 46 million people in the United States suffer from autoimmune rheumatic diseases, a number that is expected to reach 60 million by 2020. These conditions exact a significant toll on the physical health, emotional well-being, and quality of life of affected patients and are a more common cause of activity limitation than heart disease, cancer, or diabetes. Direct medical costs resulting from these rheumatic diseases are in excess of $100 billion dollars per year, with additional personal and societal costs from lost productivity raising the economic burden of these conditions exponentially. Unfortunately, traditional therapies for many of these conditions are characterized by limited effectiveness and the need for frequent monitoring.

Despite often dramatic advances in the treatment of other serious diseases, progress in developing new therapies for many autoimmune rheumatic conditions, including systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), Sjogren's syndrome, psoriasis, and scleroderma, has been slow, and the mainstays of treatment remain largely unchanged. Autoimmune diseases are particularly complex and difficult to treat, and therapies for these disorders have been only partially effective at best. Historically, treatment of autoimmune diseases has consisted primarily of methotrexate, hydroxychloroquine, and steroids, agents that provide some symptomatic relief in a portion of patients but often do not prevent inflammation-related tissue/joint damage. In addition, they may lose effectiveness over time and are associated with various problematic side effects. Recently, monoclonal antibodies for the treatment of SLE, RA, and psoriasis have become available. However, monoclonal antibodies must be injected, raising issues of patient acceptance and tolerability. Moreover, since many of these agents are associated with potentially serious side effects, their use is generally reserved for patients with moderate-to-severe disease or who have not responded to other treatments. Clearly, a new more effective treatment, preferably an oral drug, that is beneficial in a significant percentage of patients, especially those with mild-to-moderate and/or early-stage autoimmune disease, is sorely needed.

The need for new approaches is particularly pressing in the case of SLE. Until recently, no new agent for SLE had been approved in half a century, and patients have had to struggle to draw attention to their disease. As Dr. Abby Abelson, Chair of the Department of Rheumatologic and Immunologic Disease at the Cleveland Clinic commented, "I don't think there's a conspiracy here, but [lupus] just hasn't gotten a lot of funding and it hasn't gotten a lot of attention from the media." Although the monoclonal antibody, Benlysta, is now available, clinical trial results indicate that only 1 in every 11 SLE patients treated will benefit. In light of this, one FDA committee member, Dr. Lenore M. Buckley stated, "There is a need for a drug with even mild efficacy." New approaches are urgently needed for other related conditions as well, including rheumatoid arthritis (RA), Sjogren's syndrome, psoriasis, and scleroderma.